A Phase 3 Multi-Center, One-Year, Open-Label Study of Setmelanotide in Pediatric Patients Aged 2 to <6 Years of Age With Rare Genetic Causes of Obesity

RM-493-033

This is a phase 3 open-label, one-arm, clinical study to evaluate the efficacy, safety and tolerability of setmelanotide over 1 year of treatment, in pediatric patients aged 2 to \<6 years with obesity due to either biallelic variants of the POMC, PCSK1 or LEPR genes or Bardet-Biedl Syndrome (BBS).

2022-02-16

2023-09-19

2023-09-19

Completed

Early phase I

12

Key Inclusion Criteria: 1. Patients must have obesity due to either: 1. POMC, PCSK1, or LEPR deficiency, confirmed by genetic testing demonstrating biallelic variants that are interpreted as pathogenic, likely pathogenic, or of undetermined significance (VUS) by the American College of Medical Genetics and Genomics criteria (ACMG), or 2. BBS confirmed clinical and genetic diagnosis 2. Age between 2 to \<6 years at the time of informed consent 3. Obesity, defined as BMI ≥97th percentile for age and gender AND body weight of at least 15 kg at the time of enrollment. 4. Symptoms or behaviors of hyperphagia 5. Parent or guardian of study participant is able to understand and comply with the requirements of the study (including once daily \[QD\] injection regimen and all other study procedures) and is able to understand and sign the written consent/assent. Key Exclusion Criteria 1. HbA1c \>9.0% at screening 2. History of significant liver disease 3. Glomerular filtration rate (GFR) \<60 mL/min/1.73 m2 4. History or close family history of melanoma, or patient history of oculocutaneous albinism. 5. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion) 6. Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing. 7. Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide. 8. Significant hypersensitivity to any excipient in the study drug. 9. Inadequate hepatic function 10. Any other uncontrolled endocrine, metabolic or medical condition(s) known to impact body weight Other protocol defined Inclusion/Exclusion criteria may apply.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 12, 'type': 'ACTUAL'}}

2023-10-04